FDA Approves Leucovorin for Autism-Linked Cerebral Folate Deficiency Amid Expert Caution

Leucovorin, a synthetic form of vitamin B9 typically used in cancer treatment, has gained attention as a potential therapy to improve certain symptoms in children with autism, such as speech and social interaction. President Donald Trump and Health Secretary Robert F. Kennedy Jr. highlighted the drug's promise at a recent press conference, prompting the FDA to initiate approval for leucovorin calcium as a treatment for cerebral folate deficiency, a rare disorder that can mimic autism symptoms. Dr. Richard Frye, a pediatric neurologist who has studied leucovorin for two decades, notes that while leucovorin is not a cure and more research is needed, preliminary studies suggest it could benefit some autistic children by bypassing a folate transport blockage in the brain. Families of children with autism report improvements in speech and behavior after treatment, though they emphasize the drug is experimental and used alongside behavioral therapies. Experts caution that the FDA's approval process is proceeding despite a lack of large phase 3 clinical trials, underscoring the need for further research to determine optimal dosing and identify which children might benefit most. The developments highlight the intersection of emerging scientific evidence, clinical practice, and patient demand in autism treatment research.